The Child-Centered Outcomes in Practice and Research (COPR) Center of Excellence: Strengthening the Clinical Validity Evidence Base for PROMIS Measures in Chronically Ill Children

 描述(由申请人提供)：患者报告结果(PRO)仪器通过从患者的角度检查他们的感受、他们认为他们可以做什么、他们认为他们可以参与哪些类型的活动以及他们对自己的健康和生活的满意度来补充传统的临床结果。为了推进PRO评估的科学性，美国国立卫生研究院于2004年推出了患者报告结果评估信息系统(PROIS(R))。尽管PROMIS的一个关键目标是为整个生命周期的研究和临床实践提供专业知识，但关于PROMIS对患有慢性病的儿童的有效性，或者PROMIS在多大程度上检测到儿童健康中具有临床意义的变化，人们知之甚少。为了满足这些科学需求，我们建议创建以儿童为中心的实践和研究结果(COPR)卓越中心，该中心将致力于严格检查和记录PROMIS对患有慢性病的儿童的临床有效性。COPR包括三个研究项目，重点是儿科克罗恩病、慢性肾脏疾病和癌症。这些项目通过对儿童经历的幸福感(一个既包括痛苦又包括幸福的概念)、临床验证方法和基础设施的共同衡量而联系在一起。每个项目将评估儿童疼痛干预、疲劳、心理应激体验和积极情感措施的内容效度、评分的临床意义、对临床变化的反应性和纵向结构效度。COPR方法将根据PROMIS标准增加措施的心理测量和临床成熟度，并提供FDA要求的证据，以使PRO设备有资格进行临床试验。COPR团队的成员包括：一个PROMIS研究小组，制定了现有20项PROMIS儿科措施中的10项，并在过去6年中帮助领导了PROMIS计划；一个由PCORI资助的临床数据研究网络(PEDSnet)，在电子健康记录和PRO数据管理、质量评估和报告方面拥有深厚的专业知识；一个由儿科胃肠病专家组成的全国网络，正在进行PCORI资助的针对克罗恩病儿童的临床试验；一个由NIH资助的针对患有慢性肾脏疾病的儿童进行队列研究的全国儿科肾病医生网络；以及位于圣裘德儿童研究医院的全国最大的儿科肿瘤学临床中心之一。COPR基础设施将：促进其三个研究项目的协调；标准化临床验证方法和信息学资源；综合三个项目的结果；开发基于标准的仪器档案，以促进PROMIS的临床采用，并促进FDA将PROMIS措施作为临床试验终点的资格；以及支持与其他PEPR研究计划的合作。COPR经过优化，可以成功完成其三个研究项目，其基础设施可以轻松扩展以支持 信息学、统计学和管理方面的需求，以及其他多个PEPR研究计划。

 DESCRIPTION (provided by applicant): Patient-reported outcome (PROs) instruments complement conventional clinical outcomes by examining from the perspective of patients how they feel, what they perceive they can do, which types of activities they believe they can participate in, and how satisfied they are with their health and lives. To advance the science of PRO assessment, the NIH launched in 2004 the Patient Reported Outcome Measurement Information System (PROMIS(r)). Although a key objective of PROMIS is to provide PROs for research and clinical practice across the life course, there is remarkably little known about the validity of PROMIS for children with chronic diseases or the degree to which PROMIS detects clinically meaningful changes in children's health. To address these scientific needs, we propose to create the Child-Centered Outcomes in Practice and Research (COPR) Center of Excellence, which will be devoted to rigorously examining and documenting PROMIS' clinical validity for children with chronic conditions. COPR comprises three research projects focusing on pediatric Crohn's disease, chronic kidney disease, and cancer. The projects are linked by common measures of children's Experienced Well-Being (a concept that subsumes both suffering and happiness), clinical validation methods, and infrastructure. Each project will assess the content validity, clinical meaningfulness of scores, responsiveness to clinical change, and longitudinal construct validity of the PROMIS Pediatric pain interference, fatigue, psychological stress experiences, and positive affect measures. The COPR approach will increase the measures' psychometric and clinical maturation according to PROMIS standards and provide evidence required by the FDA to qualify PRO instruments for clinical trials. The COPR team integrates: a PROMIS research group that developed 10 of the 20 extant PROMIS pediatric measures and helped lead the PROMIS initiative over the past 6 years; a PCORI-funded clinical data research network (PEDSnet) with deep expertise in electronic health record and PRO data management, quality assessment and reporting; a national network of pediatric gastroenterologists embarking on a PCORI-funded clinical trial for children with Crohn's disease; a national network of pediatric nephrologists who are conducting an NIH-funded cohort study on children with chronic kidney disease; and, one of the nation's largest pediatric oncology clinical centers located at St. Jude Children's Research Hospital. The COPR infrastructure will: facilitate coordination across its three research projects; standardize clinicl validation methods and informatics resources; synthesize results across the three projects; develop standards-based instrument dossiers to enhance clinical adoption of PROMIS and facilitate FDA qualification of PROMIS measures as clinical trial end-points; and, support collaboration with other PEPR research programs. COPR is optimized to successfully accomplish its three research projects, and its infrastructure can be readily scaled to support the informatics, statistical, and administrative needs of multiple other PEPR research initiatives.